Some gene therapy treatments use modified adeno-associated viruses (AAVs) like the one shown here to deliver therapeutic genetic material into a patient’s cells. In a finding with implications for one ...
New technology enables the insertion of a large segment of DNA into a genome, potentially expanding gene therapy treatment from cancellation of disease-causing mutations to replacement of an entire ...
The US Food and Drug Administration (FDA) has released a final guidance document with immediate effect advising sponsors on ...
DMD gene therapy faces challenges in safety, efficacy, and long-term outcomes, despite FDA approval of delandistrogene moxeparvovec. Future strategies focus on novel AAV capsids, dual/triple vector ...
ICV is a commonly used neurosurgical procedure performed across multiple therapeutic settings, including gene therapyPresentation adds to body of evidence that ICV administration results in broad ...
A new gene therapy can reverse the effects of heart failure and restore heart function in a large animal model. The therapy increases the amount of blood the heart can pump and dramatically improves ...
Authorization advances Fractyl Health to a dual clinical-stage company, with Revita® in pivotal development for post-GLP-1 weight maintenance and ...
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